Manuel Carcao1,2,3, Laura Zunino1, Nancy L Young1,3,4, Saunya Dover1, Vanessa Bouskill5, Pamela Hilliard1, Victoria E Price6, Victor S Blanchette1,2,3. 1. Child Health Evaluative Sciences, The Hospital for Sick Children Research Institute, Toronto, ON. 2. Division of Hematology/Oncology, The Hospital for Sick Children, Toronto, ON, Canada. 3. Department of Pediatrics, University of Toronto, Toronto, ON, Canada. 4. School of Rural and Northern Health, Laurentian University, Sudbury, ON, Canada. 5. Department of Nursing, The Hospital for Sick Children, Toronto, ON, Canada. 6. Division of Pediatric Hematology/Oncology, Department of Pediatrics, IWK Health Centre, Dalhousie University, Halifax, NS, Canada.
Abstract
INTRODUCTION: In many countries, there is a shift from standard half-life (SHL) to extended half-life (EHL) clotting factor concentrates (CFCs). AIM: To describe the experience of switching from SHL to an EHL FVIII CFC and the impact of this on frequency of infusions, factor consumption, bleeding rates and HRQoL using the Canadian Hemophilia Kids' Life Assessment Tool (CHO-KLAT). METHODS: A retrospective chart review was conducted at a single haemophilia treatment centre in 2018 that included boys (ages: 4-18 years) with moderate/severe haemophilia A, without inhibitors, who switched from a SHL to an EHL FVIII CFC in the previous 2 years and for whom HRQoL data were available. RESULTS: The study cohort comprised 38 boys [mean (SD) age: 11.0 (3.4) years] with moderate (n = 5)/severe (n = 33) haemophilia A. The switch was associated with a 33% reduction in the number of weekly infusions from a median of 3.5 to 2.3 (P < .0001) and a 17% reduction in median FVIII consumption from 103 IU/kg/wk to 85.5 IU/kg/wk (P = .004). There was no significant change in annualized joint bleed rates or in CHO-KLAT scores. CONCLUSIONS: Despite documenting several benefits of switching to EHL FVIII (less infusions, lower factor consumption with no increase in bleeding), our study did not demonstrate any improvement in HRQoL. We conclude that either the current CHO-KLAT tool is not optimized to measure burden of treatment administration in boys with low bleed rates switching from SHL to EHL FVIII CFCs or that a reduction of 1.2 infusions/week does not result in a meaningful change in HRQoL.
INTRODUCTION: In many countries, there is a shift from standard half-life (SHL) to extended half-life (EHL) clotting factor concentrates (CFCs). AIM: To describe the experience of switching from SHL to an EHL FVIII CFC and the impact of this on frequency of infusions, factor consumption, bleeding rates and HRQoL using the Canadian Hemophilia Kids' Life Assessment Tool (CHO-KLAT). METHODS: A retrospective chart review was conducted at a single haemophilia treatment centre in 2018 that included boys (ages: 4-18 years) with moderate/severe haemophilia A, without inhibitors, who switched from a SHL to an EHL FVIII CFC in the previous 2 years and for whom HRQoL data were available. RESULTS: The study cohort comprised 38 boys [mean (SD) age: 11.0 (3.4) years] with moderate (n = 5)/severe (n = 33) haemophilia A. The switch was associated with a 33% reduction in the number of weekly infusions from a median of 3.5 to 2.3 (P < .0001) and a 17% reduction in median FVIII consumption from 103 IU/kg/wk to 85.5 IU/kg/wk (P = .004). There was no significant change in annualized joint bleed rates or in CHO-KLAT scores. CONCLUSIONS: Despite documenting several benefits of switching to EHL FVIII (less infusions, lower factor consumption with no increase in bleeding), our study did not demonstrate any improvement in HRQoL. We conclude that either the current CHO-KLAT tool is not optimized to measure burden of treatment administration in boys with low bleed rates switching from SHL to EHL FVIII CFCs or that a reduction of 1.2 infusions/week does not result in a meaningful change in HRQoL.
Keywords:
Canadian Hemophilia Outcomes Kids’ Life Assessment Tool (CHO-KLAT); EHL FVIII; health-related quality of life; pediatrics; prophylaxis; severe haemophilia A
Authors: Erik Berntorp; Kathelijn Fischer; Daniel P Hart; Maria Elisa Mancuso; David Stephensen; Amy D Shapiro; Victor Blanchette Journal: Nat Rev Dis Primers Date: 2021-06-24 Impact factor: 52.329
Authors: Victoria E Price; Saunya Dover; Victor S Blanchette; Robert J Klaassen; Mark Belletrutti; Aisha A K Bruce; Anthony K Chan; Cindy Wakefield; Manuel Carcao; Vanessa Bouskill; Nancy L Young Journal: Res Pract Thromb Haemost Date: 2021-03-27
Authors: Haowei Linda Sun; Ming Yang; Man-Chiu Poon; Adrienne Lee; K Sue Robinson; Michelle Sholzberg; John Wu; Alfonso Iorio; Victor Blanchette; Manuel Carcao; Robert J Klaassen; Shannon Jackson Journal: Res Pract Thromb Haemost Date: 2021-10-11
Authors: Marissa J M Traets; Kathelijn Fischer; Nanda Uitslager; Paul R van der Valk; Idske C L Kremer Hovinga; Lize F D van Vulpen; Roger E G Schutgens Journal: TH Open Date: 2020-11-08